Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome

por Collaud, Fanny, Bortolussi, Giulia, Guianvarc’h, Laurence, Aronson, Sem J., Bordet, Thierry, Veron, Philippe, Charles, Severine, Vidal, Patrice, Sola, Marcelo Simon, Rundwasser, Stephanie, Dufour, Delphine G., Lacoste, Florence, Luc, Cyril, Wittenberghe, Laetitia v., Martin, Samia, Le Bec, Christine, Bosma, Piter J., Muro, Andres F., Ronzitti, Giuseppe, Hebben, Matthias, Mingozzi, Federico
Publicado 2018

Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders

por El Andari, Jihad, Renaud-Gabardos, Edith, Tulalamba, Warut, Weinmann, Jonas, Mangin, Louise, Pham, Quang Hong, Hille, Susanne, Bennett, Antonette, Attebi, Esther, Bourges, Emanuele, Leborgne, Christian, Guerchet, Nicolas, Fakhiri, Julia, Krämer, Chiara, Wiedtke, Ellen, McKenna, Robert, Guianvarc’h, Laurence, Toueille, Magali, Ronzitti, Giuseppe, Hebben, Matthias, Mingozzi, Federico, VandenDriessche, Thierry, Agbandje-McKenna, Mavis, Müller, Oliver J., Chuah, Marinee K., Buj-Bello, Ana, Grimm, Dirk
Publicado 2022